Cannabidiol in Sickle Cell Disease

Phase 1

Not yet recruiting

• Conditions: Sickle Cell Disease
• Interventions: Drug: Placebo; Drug: Cannabidiol

• Registration Number: NCT06930703
• Lead Sponsor: Icahn School of Medicine at Mount Sinai

Brief Summary

Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.

Detailed Description

The researchers will conduct a randomized, double blind, placebo-controlled, study of cannabidiol in an oral formulation. Participants will be enrolled when they are not in pain crisis and have demonstrated a urine toxicology test free from cannabinoids in the past 30 days. The sample size will be 52 participants, aged ≥18, with 1:1:1:1 allocation of placebo to 3 drug doses. This is a dose finding study with a primary outcome of reduction of inflammatory cytokine TNFα.

Study Timeline

• Status Verified: 2025-04
• Study Start: 2025-04
• Study First Submitted: 2025-04-09
• Study First Submitted QC: 2025-04-09
• Last Update Submitted: 2025-04-09
• Study First Posted: 2025-04-16
• Last Update Posted: 2025-04-16
• Primary Completion: 2027-02-01
• Study Completion: 2027-02-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 52

Inclusion Criteria

• Age >18 years
• Clinical diagnosis of SCD (HbSS, HbSC, HbSβ+; Thal, HbSβ0Thal, HbS variants)
• Baseline score of 60 or lower on the ASCQ-Me 7-day pain interference domain
• If on a SCD modifying therapy (hydroxyurea, regular blood transfusions, L-glutamine, voxelotor, crizanlizumab), on stable dose for at least 3 months
• If using opioids for pain at home, on stable dose for at least 3 months
• One urine toxicology negative for cannabinoids within 30 days of randomization
• Willing to abstain from cannabis, medical and illicit, during study weeks 1 through 4 • Not pregnant or nursing
• If a woman capable of becoming pregnant, willing to use a medically accepted form of birth control for the duration of study participation. Accepted forms include oral contraception, medroxyprogesterone, contraceptive implants or patch, surgical sterilization, total abstinence.
• Able to consent for research

Exclusion Criteria

• No known intolerance to cannabinoids
• No history of psychotic episode, psychosis, or active suicidality
• No contraindication to epidiolex with attention to potential side effects, concurrent medications/substances, and concurrent medical problems, as evaluated by a physician
• Not a daily cannabis user
• No diagnosis of active substance use disorder
• No ALT>3 times the upper limit of normal

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Twice daily matching placebo
Cannabidiol 200 mg	Cannabidiol	Twice daily 200 mg cannabidiol
Cannabidiol 400 mg	Cannabidiol	Twice daily 400 mg cannabidiol
Cannabidiol 600 mg	Cannabidiol	Twice daily 600 mg cannabidiol

Primary Outcome Measures

Name	Time	Method
Tumor Necrosis Factor-alpha level	at 4 weeks	Plasma levels of tumor necrosis factor-alpha, a marker of inflammation in sickle cell disease will be collected.

Secondary Outcome Measures

Name	Time	Method
Levels of Markers of Inflammation	at 4 weeks	Levels of inflammatory markers (IL1a, IL1b, IL6, IL4 and IL10) will be collected.
Cytokines levels	at 4 weeks	Cytokines is a protein measured in blood samples to assess the body's immune and inflammatory responses to treatment. Elevated or reduced levels can indicate how the immune system is reacting and may help determine treatment effectiveness or disease progression.
Adult Sickle Cell Quality of Life Measurement System	at 4 weeks	The ASCQ-Me pain scale ranges from 0-100, with a standardized sickle cell disease population mean of 50 (standard deviation=10), where lower scores signify worse disease impact.
White Blood Cell with differential	at 4 weeks	The blood differential test measures the percentage of each type of white blood cell (WBC) that are in the blood. This test is done to diagnose an infection, anemia, or leukemia. It may also be used to monitor a condition or to see if treatment is working.
C-reactive protein level	at 4 weeks	C-reactive protein (CRP) is produced by the liver. The CRP test is a general test to check for inflammation in the body. It is not a specific test. This means it can reveal that inflammation is present somewhere in the body, but it cannot pinpoint the exact location or reason.
Tryptase levels	at 4 weeks	Tryptase is enzyme released by mast cells in the body during allergic reactions and other immune responses. Elevated levels in the blood can indicate severe allergic reactions (like anaphylaxis), mast cell disorders, or certain inflammatory conditions.
Substance P levels	at 4 weeks	Substance P is neuropeptide that functions as a neurotransmitter and acts as a key mediator of pain sensation and inflammation in the body. It plays a role in various physiological processes including stress responses, mood regulation, and neurogenic inflammation.
Patient Reported Outcome Information System (PROMIS) to measure Pain	at 4 weeks	Patient Reported Outcome Information System (PROMIS) domains for pain impact, neuropathic pain, and nociceptive pain: A standardized patient-reported measure assessing different aspects of pain experience. The measure includes subscales for pain impact (0-40), neuropathic pain (0-30), and nociceptive pain (0-30), each evaluating distinct pain mechanisms and experiences. Total score ranges from 0-100, with higher scores indicating greater pain severity and impact on daily functioning. Results are reported as T-scores (population mean=50, standard deviation=10)
Leeds Assessment of Neuropathic Symptoms and Signs pain scale (S-LANSS)	4 weeks	Leeds assessment of neuropathic signs and symptoms; The S-LANSS is a self-reported version of the Leeds Assessment of Neuropathic Symptoms and Signs pain scale. It aims to differentiate neuropathic pain (pain from nerve damage) from somatic or nociceptive pain (pain from body damage). Scores range from 0-24, with higher scores indicating greater pain.
Patient Reported Outcomes Measurement Information System (PROMIS)	at 4 weeks	The PROMIS (Patient-Reported Outcomes Measurement Information System) for domains: Anxiety, Appetite, Nausea, and Cognitive function. The PROMIS measure is a brief, computer-adapted measure of symptoms for the domains. Each is a normalized measure with a mean of 50 and standard deviation of 10. Higher scores represent increased symptoms.
Oral Morphine Equivalents (OME)	at 4 weeks	Oral Morphine Equivalents (OME)
Number of Emergency Room Visits	at 4 weeks	Number of emergency room visits
Number of Hospital Admissions	at 4 weeks	Number of hospital admissions
Number of Psychiatric Facility Utilizations	at 4 weeks	Number of psychiatric facility utilization
Columbia-Suicide Severity Rating Scale - Severity and Intensity Subscale (C-SSRS SI)	at 4 weeks	The C-SSRS rates an individual's degree of suicidal ideation (SI) on a scale, ranging from "wish to be dead" to "active suicidal ideation with specific plan and intent." The subscale identifies SI severity and intensity, which may be indicative of an individual's intent to commit suicide. C-SSRS SI ranges from 0 (no SI) to 5 (active SI with plan and intent). Higher score indicates more severity.
Prodromal Questionnaire - Brief Version (PQ-B)	at 4 weeks	The Prodromal Questionnaire Brief Version (PQ-B) is a patient questionnaire that assesses the existence of a prodromal state or fully developed psychosis. The PQ-B includes 21 items that refer to thoughts, feelings and experiences that describe various symptoms, including abnormal perception, unconventional thinking, paranoia and negative symptoms. For each item, the participant is asked to indicate whether they had experienced that phenomenon in the past month (yes/no). The total score is calculated by summing the number of "yes" responses across all items. Full range is 0-21, with higher scores indicating a greater likelihood of experiencing prodromal symptoms or fully developed psychosis

Trial Locations

Locations (1)

Icahn School of Medicine at Mount Sinai; 🇺🇸 Manhattan, New York, United States